SAN FRANCISCO—No sooner did the J.P. Morgan 44th Annual Healthcare Conference kick off when Sarepta Therapeutics (SRPT) saw its stock skid 11% after the company announced preliminary unaudited 2025 sales results that missed analyst forecasts for its marketed Elevidys® (delandistrogene moxeparvovec-rokl), which reignited the longtime issue of gene therapy safety following the deaths of three Duchenne muscular dystrophy (DMD) patients last year.
Sarepta reported total net product revenue of $369.6 million for the fourth quarter, of which about one-third ($110.4 million) was generated by Elevidys, an adeno-associated virus (AAV) gene therapy and the only gene therapy to win FDA approval as a DMD treatment. That’s down 17% from $132 million in Q3.
Sarepta Therapeutics CEO Douglas S. Ingram
Elevidys sales also fell 8% short of an analyst consensus forecast of $120.5 million cited by Sami Corwin, PhD, a biotechnology-focused healthcare analyst with William Blair, 14% below Corwin’s own forecast of $128.8 million in Q4 net product revenue, and 15% south of the $130 million estimate of Joseph P. Schwartz, senior managing director, rare diseases, and a senior research analyst with Leerink Partners.
Investors punished Sarepta with a selloff that sent the company’s shares sliding from $23.83 to $21.14. The stock bounced back somewhat over the following two days, to $22.80 at Wednesday’s close, before sliding again by Friday to $21.13 and an 11% decline for this past week.
The stock selloff came despite Sarepta beating analyst forecasts on total net product revenue, which came in at $369.6 million for Q4 and $1.86 billion for full-year 2025. The other key portion of net product revenue is sales of Sarepta’s marketed phosphorodiamidate morpholino oligomer (PMO) therapies, which also beat analyst forecasts, reaching $259.2 million for the fourth quarter and $965.6 million for all of 2025. PMOs consist of Exondys 51® (eteplirsen), Vyondys® (golodirsersen), and Amondys 45® (casimersen).
Those successes help explain why Sarepta finished 2025 with a preliminary year-end cash, cash equivalents, restricted cash, and investments balance of approximately $953.8 million, up 10% from $865.2 million as of September 30, 2025, but down 37% from $1.504 billion as of September 30, 2024.
“While we were waiting for some cannibalization of the PMO franchise from Elevidys, this has not yet materialized, as the Elevidys ramp has been impacted in recent quarters,” Schwartz wrote in his research note.
Sarepta blamed its lower-than-expected Elevidys revenue partially on the severe flu season that started around the end of 2025, as well as its need to reschedule six patient infusions from December into 2026 “for safety reasons,” CEO Douglas S. Ingram said during his presentation at the conference, held at the Westin St. Francis Hotel.
“Although Elevidys infusions were expected to be down in the fourth quarter, we view the Elevidys revenue miss as unfortunate,” Corwin wrote in a research note.
$14M–$15M in lost sales
Those six rescheduled infusions cost the company between $14 million and $15 million in sales, based on an assumed net price for Elevidys treatment of $2.4 million, Andrew Tsai, equity research analyst with Jefferies, estimated in a research note. Elevidys carries a list price or wholesale acquisition cost of $3.2 million.
For 2026, Sarepta reconfirmed its estimate of an annual sales minimum or “floor” for Elevidys of $500 million, a floor the company said it intends to exceed.
“Despite the company reiterating the anticipated annual Elevidys sales floor of $500 million,” Corwin added, “the lack of revenue guidance makes it challenging to determine if the company is seeing a true return in Elevidys demand.”
During Sarepta’s J.P. Morgan presentation and in a statement, Ingram said the company will not provide more detailed guidance to investors on 2026 Elevidys sales “until we begin to evaluate the results of our initiatives.”
Those initiatives, Ingram said, include promoting Elevidys’ benefits to boys ages 4+ with DMD to healthcare providers and patient advocates.
A 16-year-old male succumbed in March to acute liver failure after being dosed with Elevidys, the only gene therapy to win FDA approval as a DMD treatment. Three months later, a second Elevidys patient of undisclosed age died. As both patients were non-ambulatory, Sarepta halted shipments of Elevidys for non-ambulatory patients and paused the Phase III ENVISION trial (NCT05881408).
Following a third death, that of an 8-year-old Brazilian boy, the FDA demanded Sarepta pause shipments of Elevidys to ambulant patients. Sarepta initially refused before agreeing on July 21. A few days later, the FDA reversed itself, recommending that Sarepta resume Elevidys shipments to ambulant patients, after Brazilian authorities ruled out treatment with the gene therapy as a factor in the boy’s death.
The confrontation reflected a more conservative approach to regulating drugs and approval applications by Vinayak (Vinay) Prasad, MD, director of the FDA’s Center for Biologics Evaluation and Research. Prasad resigned on July 29 but returned to his FDA post on August 9.
Balancing safety, efficacy talk
“In 2025, for very good reason, we spent the bulk of our time talking about safety,” Ingram said. “It’s now time to balance that and start also talking about the absolute wealth of evidence supporting the efficacy of Elevidys and the disease-slowing nature of Elevidys. And we’re going to do exactly that. We have a really well-developed plan to do that.”
Ingram offered an outline of that plan at J.P. Morgan.
“We have significantly increased the size of our sales force for better reach. We are augmenting that with a muscular promotional campaign to support it. And we have really interesting initiatives with the patient community to accurately, thoughtfully communicate with them,” Ingram continued.
Patient advocates and healthcare providers last year joined conservative leaders in successfully persuading the FDA to step back from confronting Sarepta following the patient deaths, according to news reports, after pleading with the agency as well as Congress and President Donald Trump. The patient advocates joined conservative leaders in launching a Change.org petition that had garnered 1,565 signatures as of January 16.
“The absolutely primary initiative commercially for Elevidys in 2026 is to broadly communicate the wealth of evidence on the disease-slowing nature of Elevidys,” Ingram declared.
For example, he said, Sarepta will spend a lot of its time discussing the positive results from Parts 1 and 2 of its Phase III EMBARK trial (Study SRP-9001-301, NCT05096221). In 2023, Sarepta said Elevidys reached statistical significance on all key pre-specified secondary endpoints, including time to rise and 10-meter walk test—evidence, according to the company, of a clinically meaningful treatment benefit that was similar in magnitude and statistical significance across all age groups studied.
A year ago this month, Sarepta trumpeted positive topline results from Part 2 of EMBARK, showing that crossover-treated patients—those receiving placebo in Part 1 and crossing over at 52 weeks to be treated with Elevidys in Part 2—improved 2.34 points from baseline compared to matched external controls on the North Star Ambulatory Assessment (NSAA) 52 weeks after treatment.
Crossover-treated patients showed clinically meaningful and statistically significant functional benefit for NSAA, time to rise, and 10-meter walk/run function tests vs. a pre-specified, propensity-weighted external control group, Sarepta said, despite the crossover patients being one year older (average age 7.18 years) than those treated in Part 1 (average age 5.98 years).
“Indeed, mgmt [management] spent time emphasizing Elevidys’ previously-reported efficacy profile, suggesting that it is working to try to revitalize the narrative for the gene therapy in the face of recent benefit/risk skepticism,” Brian Abrahams, MD, managing director and global sector head, healthcare research with RBC Capital Markets, told investors, as reported by Investor’s Business Daily.
Abrahams, who also heads biotechnology equity research for RBC, added that Sarepta’s decision not to offer investors guidance on Elevidys sales “may not be well received as it may be indicative of continued uncertainty in uptake.”
“Enormous opportunity”
Sarepta and Ingram, however, reason that increased communication of past positive trial results should enable it to increase uptake by expanding Elevidys’ patient base.
“Elevidys is a tremendous therapy that has already brought a better life to over 1,100 boys and young men, and yet 80% of the addressable ambulatory population remains to be treated,” Ingram disclosed. “That is an enormous opportunity.”
As for non-ambulatory patients, Ingram said, the pathway back to dosing them would be by generating positive full 12-week safety and expression data from cohort 8 of Sarepta’s Phase I ENDEAVOR trial (also known as Study SRP-9001-103, NCT04626674), an open-label gene transfer therapy study assessing the safety of and expression of Elevidys with sirolimus in DMD patients.
“We’re executing that trial now, and you can expect those results at the very back end of this year,” Ingram said.
Leaders and laggards
Abivax (Euronext Paris and NASDAQ: ABVX) shares surged 32% from €99 ($114.78) to €130.80 ($151.65) on January 12 as of 11:58 a.m. after French news outlet La Lettre reported that Eli Lilly (NYSE: LLY) was pursuing a potential €15 billion ($17.4 billion) acquisition of the Paris-based inflammatory disease drug developer. According to the report, Lilly was awaiting confirmation from France’s Ministry of Economics, Finance, and Industrial and Digital Sovereignty about whether an acquisition would be subject to French foreign investment control procedures. La Lettre reported that Lilly became interested in Abivax last year after it reported dazzling Phase III data for its lead pipeline candidate obefazimod in adults with moderately to severely active ulcerative colitis, sparking a six-fold surge in Abivax’s stock price. The finance ministry denied to Reuters having any contact with Lilly, which declined comment on business development activity. “The rumors, speculations, we have no control over those,” Abivax CEO Marc de Garidel told BiotechTV. Shares fell for the rest of the week, trading between €100 and €113 (approximately $116-$131).
ImmunityBio (NASDAQ: IBRX) shares rocketed 83% over two days, leaping 31% Thursday from $3.02 to $3.95, then jumping another 40% to $5.52 Friday. Two positive developments drove the surge: ImmunityBio said Wednesday that the Saudi FDA approved its marketed cancer drug Anktiva® (nogapendekin alfa inbakicept-pmln) plus immune checkpoint inhibitors for adults with metastatic non-small cell lung cancer who failed standard therapies, marking the company’s first global approval for the indication and first for subcutaneous administration. On Thursday, the developer of cancer and infectious disease treatments announced a 700% year-over-year surge in full-year preliminary net product revenue, which rose to approximately $113 million from $14.15 million in 2024. Driving that surge was a ~750% increase in unit sales volume growth for Anktiva in 2025 vs. 2024. Anktiva is the first FDA-approved immunotherapy for non-muscle invasive bladder cancer CIS designed to activate NK cells, T cells, and memory T cells for a long-duration response.
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